First in human clinical study of a novel drug JNT-517 to assess its safety and tolerability in healthy volunteers
- Conditions
- PhenylketonuriaMetabolic and Endocrine - Metabolic disordersHuman Genetics and Inherited Disorders - Other human genetics and inherited disorders
Recruitment & Eligibility
- Status
- Completed
- Sex
- All
- Target Recruitment
- 72
1. Males and females 18 to 55 years of age, inclusive.
2. Medically healthy with no clinically significant medical history, physical examination, laboratory results, vital signs, or ECGs at Screening and Day 1.
3. Body mass index (BMI) of 18-40 kg/m2 and total body weight >50 kg.
4. Non-smoker for at least 2 weeks prior to dosing and willing to abstain during the study.
5. Females of childbearing potential involved in any sexual intercourse that could lead to pregnancy: the participant must agree to use two highly effective contraceptive methods from Screening until at least 1 week after the last study drug administration.
a. Female participants of childbearing potential must have a negative serum pregnancy test at Screening and a negative urine pregnancy test by Day 1.
6. Females not of childbearing potential or postmenopausal defined as follows:
a. Have had surgical sterilization (hysterectomy, bilateral oophorectomy, or bilateral salpingectomy)
b. Have had amenorrhea for minimum of 1 year with confirmation by levels of follicle stimulating hormone testing
7. For male participants involved in any sexual intercourse that could lead to pregnancy, participant must agree to use highly effective contraceptive methods from Day 1 until at least 12 weeks after the last study drug administration.
Note: No restrictions are required for males who have undergone a documented vasectomy at least 4 months prior to Screening. If vasectomy procedure is not documented or was performed less than 4 months prior to Screening, males must follow the same contraception as for non-vasectomized participants.
8. Participants with psychiatric illness must be well-controlled for last 6 months prior to the Screening visit and if on medication, on stable medications for the last 3 months.
9. Capable of giving signed informed consent and ability to comply with study procedures.
All Parts:
1. Any acute or chronic medical condition that would prevent the participant from complying with the procedures or place the participant at risk if they participate in the study.
2. Positive for hepatitis B or C or human immunodeficiency virus.
3. Any history of malignancy in the last 5 years, excluding non-melanoma skin cancer.
4. Any history of liver disease.
5. Any surgical or medical conditions that may affect study drug absorption, distribution, metabolism, or excretion.
6. Creatinine clearance <90 mL/min by Cockcroft-Gault formula.
7. Participation in another investigational drug trial within 30 days or, if known, 5 half-lives of the investigational drug (whichever is longer).
8. Alcohol consumption within 5 days of randomization and/or unwilling to abstain during the study.
9. Use of any medications that are inhibitors or inducers of cytochrome P450 (CYP)3A4 or inhibitors of the transporter P-glycoprotein (P-gp) within 4 weeks prior to randomization and unwilling and/or unable to avoid these medications throughout the treatment duration.
10. Use of any medication that is a substrate of CYP3A4, or a substrate of the transporters P-gp, breast cancer resistance protein (BCRP), organic anion transporter 3 (OAT3), multidrug and toxin extrusion (MATE)1, or MATE2-K within 4 weeks prior to randomization and unwilling and/or unable to avoid these medications throughout the treatment duration.
11. History of drug/alcohol abuse in the last year.
12. Inability to tolerate oral medication.
13. Allergy to JNT-517 or any component of the investigational product.
14. Given >50 mL of blood or plasma within 30 days of Screening or >500 mL of blood or plasma within 60 days of Screening.
15. Any of the following laboratory values at the Screening visit:
• Alanine aminotransferase or aspartate aminotransferase values >upper limit of normal (ULN)
• Total bilirubin >ULN
• Haemoglobin <11.0 g/dL (<110.0 g/L)
• White blood cell count <4.5 × 10 9/L (<4500/mm3)
• Platelet count <150 × 10 9/L (<150,000/mm3)
16. Smoker (defined as an individual who has used nicotine-containing products, including cigarettes and e-cigarettes) within the last 2 weeks prior to dosing.
17. Positive for cotinine or drug screen.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method